Science Lab


Gene therapy

 Gene therapy is a revolutionary approach to treating genetic diseases, where a one-time treatment can provide a lifetime of benefits. Instead of treating just the symptoms, gene therapy fixes the disease at the source by replacing the defective gene with a healthy copy.

A healthy copy of the patient's defective gene is loaded into a virus that has been stripped of its own DNA.

Trillions of viruses, each containing a healthy copy of the gene, are injected into the patient's spinal fluid. 

The viruses bind to cells in the patient's spinal cord and brain and deliver healthy gene's to the cell's nucleus.

how it works

In the case that a gene changes—also known as mutating—in a way that causes Leigh syndrome, gene therapy may be able to help. Gene therapy is the introduction, removal or change in genetic material—specifically DNA or RNA—into the cells of a patient to treat a specific disease. The transferred genetic material changes how a protein—or group of proteins—is produced by the cell.

Source: American Society of Gene & Cell Therapy


Since the founding of the Cure SURF1 Foundation in 2018, we have collaborated with several organizations to develop transformative gene therapy treatments for SURF1 Leigh Syndrome.